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Completed

NCT Number: NCT07415083

A Study of the Safety and Effectiveness of Eltrombopag in Combination With Immunosuppressive Therapy (IST) in Korean Pediatric Patients With Treatment Naïve Severe Aplastic Anemia

The aim of this study was to investigate the safety, tolerability, and effectiveness of eltrombopag in combination with immunosuppressive therapy (IST) in real world practice in pediatric patients with severe aplastic anemia (sAA) in Korea. The study used data over a 6-month period extracted from patients' medical charts (electronic medical records) starting from the initiation of eltrombopag treatment.

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Key information

Conditions

Aplastic Anemia Anemia Anemia, Aplastic Bone Marrow Diseases Bone Marrow Failure Disorders Hematologic Diseases Hemic and Lymphatic Diseases

Age range

2 year–17 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Novartis

East Hanover, New Jersey, 07963, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Male or female pediatric patients aged ≥2 years and <18 years at the time of initiating eltrombopag and IST treatment.
  • Subjects diagnosed with sAA and treated with eltrombopag in combination with IST as first-line therapy, based on the physician's decision in accordance with locally approved prescribing information at the time of data collection.
  • Subjects whose analytical index date (start date of eltrombopag administration) was at least 6 months prior to the data extraction date.

Exclusion criteria

None.

Treatment and study plan

Primary outcomes

  1. Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Time frame: Up to 6 months

    Incidence of all AEs/adverse drug reactions (ADRs), SAEs/serious adverse drug reactions (SADRs), and unexpected AEs/ADRs.

Secondary outcomes

  1. Overall Response Rate (ORR)

    Time frame: 6 months

    ORR includes complete response (CR) and partial response (PR).

    CR was defined as meeting all three of the following criteria:

    • Absolute neutrophil count (ANC) > 1.0 × 10^9/L
    • Platelet count > 100 × 10^9/L
    • Hemoglobin > 100 g/L

    PR was defined as hematologic improvement that did not meet the criteria for CR, but also did not meet the criteria for sAA.

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study title

Retrospective Chart Review of Safety and Effectiveness of Eltrombopag in Combination With Immunosuppressive Therapy (IST) in Korean Pediatric Patients With Treatment Naïve Severe Aplastic Anemia

Important dates

Study start
2023
Primary completion
2025
Study completion
2025
First posted
Feb 17, 2026
Registry last updated
Feb 19, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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